A Look to Future Directions in Gene Therapy Research for Monogenic Diseases (2006)
Matthew H. Porteus, Jon P. Connelly, Shondra M. Pruett
The concept of gene therapy has long appealed to biomedical researchers and clinicians because it promised to treat certain diseases at their origins. In the last several years, there have been...
Efficient Gene Targeting Mediated by Adeno-Associated Virus and DNA Double-Strand Breaks (2003)
Porteus, Matthew H., Cathomen, Toni, Weitzman, Matthew D., Baltimore, David
Gene targeting is the in situ manipulation of the sequence of an endogenous gene by the introduction of homologous exogenous DNA. Presently, the rate of gene targeting is too low for it to be broadly...
Efficient Gene Targeting Mediated by Adeno-Associated Virus and DNA Double-Strand Breaks
Porteus, Matthew H., Cathomen, Toni, Weitzman, Matthew D., Baltimore, David
Gene targeting is the in situ manipulation of the sequence of an endogenous gene by the introduction of homologous exogenous DNA. Presently, the rate of gene targeting is too low for it to be broadly...
Durai, Sundar, Mani, Mala, Kandavelou, Karthikeyan, Wu, Joy, Porteus, Matthew H., Chandrasegaran, Srinivasan
Custom-designed zinc finger nucleases (ZFNs), proteins designed to cut at specific DNA sequences, are becoming powerful tools in gene targeting—the process of replacing a gene within a genome by...
A Look to Future Directions in Gene Therapy Research for Monogenic Diseases
Porteus, Matthew H, Connelly, Jon P, Pruett, Shondra M
The concept of gene therapy has long appealed to biomedical researchers and clinicians because it promised to treat certain diseases at their origins. In the last several years, there have been...
Efficient Gene Targeting Mediated by Adeno-Associated Virus and DNA Double-Strand Breaks
Porteus, Matthew H., Cathomen, Toni, Weitzman, Matthew D., Baltimore, David
Gene targeting is the in situ manipulation of the sequence of an endogenous gene by the introduction of homologous exogenous DNA. Presently, the rate of gene targeting is too low for it to be broadly...
Durai, Sundar, Mani, Mala, Kandavelou, Karthikeyan, Wu, Joy, Porteus, Matthew H., Chandrasegaran, Srinivasan
Custom-designed zinc finger nucleases (ZFNs), proteins designed to cut at specific DNA sequences, are becoming powerful tools in gene targeting—the process of replacing a gene within a genome by...
A Look to Future Directions in Gene Therapy Research for Monogenic Diseases
Porteus, Matthew H, Connelly, Jon P, Pruett, Shondra M
The concept of gene therapy has long appealed to biomedical researchers and clinicians because it promised to treat certain diseases at their origins. In the last several years, there have been...
Potts, Patrick Ryan, Porteus, Matthew H, Yu, Hongtao
The structural maintenance of chromosomes (SMC) family of proteins has been implicated in the repair of DNA double-strand breaks (DSBs) by homologous recombination (HR). The SMC1/3 cohesin complex is...
Submitted to the Program in Neurosciences and the Committee on Graduate Studies of Stanford University.